How Do Members of the Duchenne and Becker Muscular Dystrophy Community Perceive a Discrete-Choice Experiment Incorporating Uncertain Treatment Benefit? An Application of Research as an Event

Bridges, John F.P.; Tsai, Jui-Hua; Janssen, Ellen; Crossnohere, Norah L.; Fischer, Ryan; Peay, Holly

doi:10.1007/s40271-018-0330-8

Cited by 14 publications

(22 citation statements)

References 38 publications

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“…Fifth, this survey only met two out of three acceptability measures using the 75% threshold. 37 Less than 75% of participants agreed or strongly agreed that the choice tasks were easy to answer, which is also lower than other studies that have used this scale. 37,52…”

Section: Discussionmentioning

confidence: 79%

“…In accordance with previous publications, 75% of participants needed to answer "agree" or "strongly agree" for each acceptability question to be met. 37 The survey also asked about demographic information and disease history. Participants were not compensated for completing the survey.…”

Section: Participants and Survey Administrationmentioning

confidence: 99%

“…Most participants (77%) strongly agreed or agreed that the DCE choice tasks were easy to understand, 54% strongly agreed that they were easy to answer, and 89% of participants strongly agreed they answered the questions consistent with their preferences, meeting the threshold for two out of three acceptability questions. 37 Of 114 participants, 102 (89.4%) completed all choice tasks. No significant difference was identified between those who did and did not complete all choice tasks for all observed characteristics.…”

Section: Participantsmentioning

confidence: 99%

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<p>Analysis of Patient Preferences in Lung Cancer – Estimating Acceptable Tradeoffs Between Treatment Benefit and Side Effects</p>

Janssen¹,

Dy²,

Meara³

et al. 2020

PPA

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Objective: Increased treatment options and longer survival for lung cancer have generated increased interest in patient preferences. Previous studies of patient preferences in lung cancer have not fully explored preference heterogeneity. We demonstrate a method to explore preference heterogeneity in the willingness of patients with lung cancer and caregivers to trade progression-free survival (PFS) with side effects. Patients and Methods: Patients and caregivers attending a national lung cancer meeting completed a discrete-choice experiment (DCE) designed through a collaboration with patients. Participants answered 13 choice tasks described across PFS, short-term side effects, and four long-term side effects. Side effects were coded as a one-level change in severity (none-mild, mild-moderate, or moderate-severe). A mixed logit model in willingness-to-pay space estimated preference heterogeneity in acceptable tradeoffs (time equivalents) between PFS and side effects. The study was reported following quality indicators from the United States Food and Drug Administration's patient preference guidance. Results: A total of 87 patients and 24 caregivers participated in the DCE. Participants would trade 3.7 month PFS (95% CI (CI): 3.3-4.1) for less severe functional long-term treatment side effects, 2.3 months for less severe physical long-term effects (CI: 1.9-2.8) and cognitive long-term effects (CI: 1.8-2.8), 0.9 months (CI: 0.4-1.4) for less severe emotional long-term effects, and 1.8 months (CI: 1.4-2.3) for less severe short-term side effects. Most participants (90%) would accept treatment with more severe functional long-term effects for 8.4 additional month PFS. Conclusion: Participants would trade PFS for changes in short-term side effects and longterm side effects, although preference heterogeneity existed. Lung cancer treatments that offer less PFS but also less severe side effects might be acceptable to some patients.

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Section: Discussionmentioning

confidence: 79%

Section: Participants and Survey Administrationmentioning

confidence: 99%

Section: Participantsmentioning

confidence: 99%

See 1 more Smart Citation

<p>Analysis of Patient Preferences in Lung Cancer – Estimating Acceptable Tradeoffs Between Treatment Benefit and Side Effects</p>

Janssen¹,

Dy²,

Meara³

et al. 2020

PPA

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“…A Likert-type scale was also used to evaluate five dimensions of the caregiver’s personality, including optimistic, health-conscious, risk-taker, numbers-savvy, and self-control. Caregivers also completed standard debriefing questions regarding the DCE, 27 evaluating their experience with the DCE by indicating their agreement with three statements along a Likert-type scale, including the following: 1) I found it easy to understand the questions; 2) I found it easy to answer the questions; 3) My answers showed my real preferences.…”

Section: Methodsmentioning

confidence: 99%

Measuring Meaningful Benefit-Risk Tradeoffs to Promote Patient-Focused Drug Development in Prader-Willi Syndrome: A Discrete-Choice Experiment

Tsai

Crossnohere

Strong

et al. 2021

MDM Policy & Practice

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Background. Prader-Willi syndrome (PWS) is a rare neurodevelopmental disorder causing quality of life impairments such as insatiable hunger (hyperphagia) and obesity. We explored caregivers’ willingness to assume treatment risk in exchange for reduced hyperphagia according to a PWS-validated observer-reported outcome measure. Methods. We partnered with PWS patient organizations to develop a discrete-choice experiment exploring caregivers’ benefit-risk tradeoffs for emerging PWS treatments. The treatment benefit was a reduction in hyperphagia (as measured by a 0-, 5-, or 10-point change on the Hyperphagia Questionnaire for Clinical Trials [HQ-CT]). Treatment risks included weight gain (none, 5%, 10%), added risk of skin rash (none, 10%, 20%), and risk of liver damage (none, 1 in 1000, 10 in 1000). Preference models were estimated using mixed logistic regression and maximum acceptable risk. We explored differences in preferences across familial caregivers of patients with and without hyperphagia. Results. Four hundred sixty-eight caregivers completed the online survey. The majority of caregivers reported that patients experienced hyperphagia (68%) and half of patients experienced obesity (52%). Caregivers of patients without hyperphagia were willing to accept greater weight gain (16.4% v. 8.1%, P = 0.004) and a higher risk of skin rash (11.7% v. 6.2% P = 0.008) as compared to caregivers of patients with hyperphagia. Caregivers of patients with hyperphagia would accept a higher risk of liver damage as compared to caregivers of patients without hyperphagia (11.9 out of 1000 v. 6.4 out of 1000, P = 0.04). Conclusions. This research demonstrates that caregivers are willing to accept risk in exchange for a five-point improvement on the HQ-CT, a smaller marginal improvement than had been previously classified as meaningful. Patient experience with hyperphagia is a modifier in how much risk caregivers will accept.

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“…After completing each preference elicitation method, participants will be asked to score the level of understanding and agreement of the questionnaire using a Likert-type scale ( Table 1 ). Similar questionnaires have proven to be informative when analyzing study feasibility and heterogeneity of preferences and will provide additional information to answer the methodological research question to compare the three different preference methods 33 , 34 .…”

Section: Study Protocolmentioning

confidence: 99%

A study protocol for quantifying patient preferences in neuromuscular disorders: a case study of the IMI PREFER Project

Jimenez-Moreno¹,

Pinto²,

Levitan³

et al. 2020

Wellcome Open Res

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Objectives: Patient preference studies are increasingly used to inform decision-making during the medical product lifecycle but are rarely used to inform early stages of drug development. The primary aim of this study is to quantify treatment preferences of patients with neuromuscular disorders, which represent serious and debilitating conditions with limited or no treatment options available. Methods: This quantitative patient preferences study was designed as an online survey, with a cross-over design. This study will target two different diseases from the neuromuscular disorders disease group, myotonic dystrophy type 1 (DM1) and mitochondrial myopathies (MM). Despite having different physio-pathological pathways both DM1 and MM manifest in a clinically similar manner and may benefit from similar treatment options. The sample will be stratified into three subgroups: two patient groups differentiated by age of symptom onset and one caregivers group. Each subgroup will be randomly assigned to complete two of three different preference elicitation methods at two different time points: Q-methodology survey, discrete choice experiment, and best-worst scaling type 2, allowing cross-comparisons of the results across each study time within participants and within elicitation methods. Additional variables such as sociodemographic, clinical and health literacy will be collected to enable analysis of potential heterogeneity. Ethics and Dissemination: This study protocol has undergone ethical review and approval by the Newcastle University R&D Ethics Committee (Ref: 15169/2018). All participants will be invited to give electronic informed consent to take part in the study prior accessing the online survey. All electronic data will be anonymised prior analysis. This study is part of the Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle (IMI-PREFER) project, a public-private collaborative research project aiming to develop expert and evidence-based recommendations on how and when patient preferences can be assessed and used to inform medical product decision making.

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How Do Members of the Duchenne and Becker Muscular Dystrophy Community Perceive a Discrete-Choice Experiment Incorporating Uncertain Treatment Benefit? An Application of Research as an Event

Cited by 14 publications

References 38 publications

<p>Analysis of Patient Preferences in Lung Cancer – Estimating Acceptable Tradeoffs Between Treatment Benefit and Side Effects</p>

<p>Analysis of Patient Preferences in Lung Cancer – Estimating Acceptable Tradeoffs Between Treatment Benefit and Side Effects</p>

Measuring Meaningful Benefit-Risk Tradeoffs to Promote Patient-Focused Drug Development in Prader-Willi Syndrome: A Discrete-Choice Experiment

A study protocol for quantifying patient preferences in neuromuscular disorders: a case study of the IMI PREFER Project

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